A MEDICAL breakthrough in which scientists correct a faulty piece of DNA that causes deadly heart disease to run in families will help pave the way for pioneering genetic research in Scotland, researchers claim.
US and South Korean scientists have successfully freed embryos of a piece of corrupted of faulty DNA that causes a congenital heart disease for the first time.
Mutations were corrected by a groundbreaking biological tool called CRISPR-Cas9, which cuts away the enzyme which causes the defect.
It is the first time the controversial technique has been used on fertilised human embryos as opposed to embryonic stem cells.
However, the study is still in the early experimental stage and would not be made to create babies with the genetic modification, which would be illegal both in the US and the UK.
Dr Alasdair MacKenzie, Reader at the Institute of Medical Sciences, at Aberdeen University, works with CRISPR in his research.
He said it was an “exciting” development and shows the potential of using this kind of research in Scotland.
He said: “There’s very few labs so far that have gone ahead and started to even consider this.
“There’s many ethical issues to get over before people start... But essentially the technology’s all there.”
He said that the study could be applied to research into Huntington’s Disease where the CRISPR technique could potentially work particularly well against the ‘triplet repeats’ in the gene coding of condition.
He added: “I think the ethics are absolutely well worth discussing, but you’ve got to balance it out with people suffering and dying of horrible diseases and is it ethical to allow someone to die of a horrible disease if there was a way to treat the disease at some point?”
Dr Alasdair MacKenzie urged caution, adding that more needed to be known about one side-affect, where CRISPR may go “off-target.” He said: “We need to be aware of this before we start going down the road too enthusiastically.”
Teams at the Oregon Health and Science University (OHSU) and the Salk Institute in the US, along with the Institute for Basic Science in South Korea, focused on hypertrophic cardiomyopathy, which affects one in every 500 people.
In the study, described in the journal Nature, the genetic repair happened during conception.
It did not work all the time, but 72 per cent of embryos were free from disease-causing mutations.
A leading member of the team hinted that first steps towards bringing the treatment to patients could take place in the UK under the direction of the fertility regulator the Human Fertilisation and Embryology Authority (HFEA). UK regulations are more liberal than the US.
Dr Shoukhrat Mitalipov, from Oregon Health and Science University (OHSU) in Portland, said: “Maybe .. (the) HFEA might take a lead on this, but I’m quite sure before these clinical trials can go on they have to go through, I believe, Parliament to change a law. So there is still a long road ahead, particularly if you want to do it in a regulatory way.”
British experts have called research”remarkable” and a “major advance.” but tempered their response with words of caution.
However, Dr David King, director of the Human Genetics Alert, which opposes all tampering with the human genome, said there should be a global ban on creating cloned or genetically modified (GM) babies. He said “If irresponsible scientists are not stopped, the world may soon be presented with a fait accompli of the first GM baby.”
“We call on governments and international organisations to wake up and pass an immediate global ban on creating cloned or GM babies, before it is too late.”
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